Convergence: Regulators, experts weigh fixes for orphan device challenges

PITTSBURGH — Regulators and experts sketched the global landscape for orphan medical device development and suggested solutions to common practical challenges during a session at RAPS Convergence 2025 on Thursday.

Mathias Fink, senior managing consultant at the AKRA Team consultancy in Germany, highlighted differences in the global definition what qualifies as an orphan device and therefore is deserving of incentives for development.

Per the US humanitarian use device (HUD) program, an orphan device is intended to treat, diagnose or prevent a condition affecting up to 8,000 people in the US on an annual basis. It must not expose patients to unreasonable or significant risk and probable benefits must outweigh risk of injury.

In the European Union (EU), orphan devices are aimed at sub-populations with fewer than 12,000 EU patients per year. The EU specifies that there be insufficient alternatives or that the product offers benefits over available options.

Japan’s program is more liberal – orphan products are intended for use in a population of under 50,000 in areas of high unmet medical need where there are no suitable alternatives.

“Looking at the much smaller number of people living in Japan compared to the US or the European Union, I think it gives them more flexibility,” Fink said.

The presenters addressed challenges for making use of orphan device programs and offering greater access to vulnerable patient populations, touching on profitability, and requirements to collect postmarketing data.

“A limited number of devices that is used also means that the manufacturer will not earn a lot of money – and that usually doesn’t justify really putting the effort in designing and developing a device,” said Bianca Lutters, head of clinical affairs, at the QServe Group consultancy in the Netherlands.

“What the FDA has done with the HDE program, but also with other initiatives, is made it easier for manufacturers from a financial perspective to get there,” Lutters said. “So, it's not only a program to get the devices on the market with some advantages from a review perspective, but also some financial incentives.”

Developing devices for use in children has been a perennial challenge, as the patients are vulnerable and trials are difficult to conduct, leaving regulators struggling to strike the right balance between safety and access.

But adult data is highly transferable and the risks are the same as in children, so that’s very helpful, commented Richard Holborow, head of clinical compliance at the British Standards Institute (BSI). Holborow stressed the importance of balance among notified bodies in the EU, with the health of pediatric and rare disease populations in mind.

“Notified bodies need to be pragmatic about making sure that we don't start contraindicating in pediatric populations, because maybe the danger isn't there,” said Holborow, who is also involved with the working groups of the EU’s Medical Device Coordination Group (MDCG).

Alberto Gañan Jimenez, PhD, head of committees and quality assurance at the European Medicines Agency (EMA), noted that the European Commission has developed a new expert panel to provide guidance on medical devices and in vitro diagnostics (IVDs) focused on pediatrics and rare diseases. A pilot program started in 2024 and is set to continue through 2025. (RELATED: EU expert group established for pediatric and rare disease devices, Regulatory Focus 9 July 2025)

So far, he said, five products have been considered in the pilot program: three used in the circulatory system, one in glioblastoma and one in pediatric scoliosis.

For orphan devices in general, collecting postmarketing data is a challenge for manufacturers.

“Well-designed surveys with patient-reported outcome measures are useful and helpful in these scenarios, and that's usually lower cost and can also be implemented by a manufacturer,” Holborow said.

Notified bodies are looking for a structured protocol behind these activities to provide assurance that data are free from bias, are relevant and clinically meaningful, Holborow added.

Some manufacturers have worked with national registries to capture data for subsets of populations and where national registries don’t exist, it’s perfectly fine for manufacturers to generate its own registry after a device is placed at a hospital to capture data and feed it back, Holborow advised.

There are also registries targeted at particular interventions, involving a family of devices, and patient and clinical groups that could provide useful data, said Donal O'Connor, MD, clinical manager at the Health Products Regulatory Authority (HPRA) in Ireland and co-chair of the European Commission MDCG task force on orphan devices.

“We've had discussions with the rare disease community – the clinical community – in Europe, to inform them, if you want devices to treat your patients and diagnose your patients, you may have powerful data and you may be able to collaborate with the manufacturers,” said O’Connor. “And some of the clinical societies may be open to just add a couple of data points that will be relevant for your PMCF [postmarket clinical follow-up.”

Organizations representing patients with rare diseases may be really willing to “go the extra mile,” panelist Lutters weighed in.

“They may help in getting patients engaged and enthusiastic to sign informed consent for their data to be collected,” she explained.

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Convergence: Regulators, experts weigh fixes for orphan device challenges

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